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In Vivo Therapeutic Platform Based on RNA Base Editing

The RNA base editing platform aims to develop therapies for Hurler syndrome, the most severe subtype of mucopolysaccharidosis type I which include diseases related to liver, muscles and nerve system, etc. based on the RNA single-base editing technology LEAPER.

LEAPER’s purpose is to edit RNA programmatically through endogenous ADAR (adenosine deaminase acting on RNA, i.e. the RNA adenosine deaminase).


• A Short Oligo RNA (<100nt)
• Versatility: delivered via Plasmid, Virus or LNP
• Reversibility: edit RNA, not DNA
• Robust efficiency and duration
• High precision
• No requirement of exogenous protein

Unmet Needs

Significant number of single-gene genetic diseases caused by single point mutation, of which a significant portion is G>A; Current ERT’s cost is high and with high ADA risk

Therapeutic Approach

Using an RNA molecule of <100nt to edit the mutant RNA, specifically converting significant portion of G>A mutation back to I/G, resulting in expression of wild type, endogenous protein

Papers & Presentations        

- In July 2019, paper published in Nature Biotechnology

- In May 2020, EdiGene presented data on its lead therapeutic program for patients with the most severe form of mucopolysaccharidosis type I (MPS I), known as Hurler syndrome, at the 23rd American Society of Gene & Cell Therapy (ASGCT) Annual Meeting